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  • Frances Fitzgerald

EU and Irish Stakeholders Join Forces to Discuss Access to Rare Disease Medicines

Updated: Apr 17, 2023

Press Statement from Frances Fitzgerald MEP

Friday 1st April 2022

EU and Irish Stakeholders Join Forces to Discuss Access to Rare Disease Medicines

Dublin MEP Frances Fitzgerald today (Friday) hosted a webinar focusing on finding solutions to the challenges relating to rare diseases in Ireland and the EU.

Speaking after the discussion, Frances Fitzgerald MEP said: “What came across so clearly throughout the course of our discussion today was that rare diseases are in fact not rare, rather they are collectively common. We must all do what we can to ensure a greater focus is placed on rare diseases both in Ireland and at EU level, where I have no doubt there is a lot we can learn from each other.”

Guest speaker Pádraig O’Sullivan TD said: “Ireland is a mixed bag when it comes to providing appropriate care for rare disease patients. We developed a rare disease plan in 2014 and have a strong biopharmaceutical presence in Ireland. That being said, we have low access to treatments for patients, low levels of good quality data and low clinical trial participation. Sadly, we are also waiting for a successor strategy to the rare disease plan despite it being a commitment in the Programme for Government. I hope that we can work to bring this piece of work forward.

The event addressed ongoing legislative initiatives related to rare diseases including:

  • The Private Members Bill currently progressing through the Oireachtas under the sponsorship of Fianna Fáil TD, Deputy Pádraig O’Sullivan – the Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2021 – which aims to establish separate and appropriate assessment criteria for the consideration of reimbursement of orphan medicinal products by the HSE;

  • The European Commission’s ongoing revision of the Orphan Medicinal Products Regulation which deals with designation of OMPs (Orphan Medicinal Products) and incentives to encourage their research and development by the pharmaceutical industry.

  • This webinar focused on opportunities to improve the situation for rare disease patients in Ireland. Some of the solutions proposed over the course of the discussion included:

  • Reform of the Irish drug assessment and reimbursement process to make it time bound and predictable on the basis that the current system is not fit for purpose.

  • Publication of the Mazars review of the governance structures around the HSE’s drug reimbursement process, submitted to the Department of Health in January 2020.

  • Expansion of the newborn screening programme.

  • Urgent development of a national genomics and genetics strategy.

  • Rapid development of health data infrastructural capacity.

  • Reallocation of savings made by the HSE through rebates from the pharmaceutical industry to fund access to innovative medicines.

  • More fluid exchange of patient data between European Member States.

  • Prioritisation of the development of the national rare disease plan successor strategy as per the Programme for Government.

  • More frequent meetings of the Rare Disease Technology Review Committee which facilitates the incorporation of patient and clinician perspectives into reimbursement decisions.

  • Guarantee of a rare disease lens for the upcoming Health Information and Patient Safety Bill currently being drafted.

  • Relocation of the rare disease patient group point of contact in the Department of Health from the Office of the Chief Medical Officer to a unit of the Department of Health more focused on research or European policy coordination.

  • Establishment of a cross-party parliamentary Oireachtas group and/or Committee on rare disease.


Note to Editor


HOST: Frances Fitzgerald MEP

DATE: Friday, 1st April 10.00 – 11.30 (GMT)


Padraig O’Sullivan TD

Sponsor of the Health (Pricing and Supply of Medicinal Goods) (Amendment) Bill 2021

Mr. Florian Schmidt

Deputy Head of Unit – Pharmaceutical Unit – DG Santé, European Commission

Rachel Finnegan

Senior Director of Government Affairs EMEA at BioMarin

Bernadette Gilroy

Patient Advocate and Chairperson of PKU Association of Ireland

Derick Mitchell

Chief Executive Officer of IPPOSI (Irish Platform for Patient Organisations, Science and Industry)


Conor Glynn



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